Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Thomas O. Crawford, Laurent Servais, Eugenio Mercuri, Heike Kölbel, Nancy L. Kuntz, Richard S. Finkel, Jena M. Krueger, Kaitlin Batley, Sally Dunaway Young, Jing L. Marantz, Guochen Song, Bert Yao, Guolin Zhao, José Rosselló, Giridhar Tirucherai, Elena Mazzone, Russell J. Butterfield, Marta Gómez García de la Banda, A. Seferian, Valeria Sansone, Liesbeth De Waele, W. Ludo van der Pol, C. Cancès, Astrid Pechmann, Basil T. Darras, Adelie Navas Nazario, Anne M. Connolly, Carolina Tesi Rocha, Chamindra Laverty Rady, Craig M. Zaidman, Crystal M. Proud, H. Phan, Jeffrey Statland, Jennifer M. Kwon, Jessica Nance, Julie Parsons, Katherine D. Mathews, Leigh Ramos‐Platt, Michael S. Cartwright, Randal Richardson, R. H. Fryer, Sabrina W. Yum, Saunder Bernes, Seth J. Perlman, Claudio Bruno, Riccardo Masson, Sonia Messina, Astrid Blaschek, Dorothea Holzwarth, Barbara Steinborn, Katarzyna Kotulska, Maria Mazurkiewicz‐Bełdzińska, Aurore Daron, Nicolas Deconinck, Jean‐Baptiste Davion, Anne‐Marie Childs, Giovanni Baranello, A. Nascimento Osorio, Inmaculada Pitarch Castellano